Surgical management of pediatric hepatocellular carcinoma: An analysis of the National Cancer Database

Ziogas, Ioannis A.; Benedetti, Daniel J.; Matsuoka, Lea; Izzy, Manhal; Rauf, Muhammad Abdur; Pai, Anita K; Bailey, Christina E.; Alexopoulos, Sophoclis P.

doi:10.1016/j.jpedsurg.2020.06.013

Cited by 13 publications

(23 citation statements)

References 20 publications

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“…Studies using data from the National Cancer Database (NCDB) and the Surveillance, Epidemiology, and End Results (SEER) database have shown that LT is associated with better outcomes compared to partial hepatectomy for pediatric HCC [ 9 , 10 ]. Small case series from reference centers have shown that LT can lead to favorable long-term outcomes for pediatric HCC even beyond of the Milan criteria [ 11 , 12 ].…”

Section: Introductionmentioning

confidence: 99%

Liver Transplantation for Pediatric Hepatocellular Carcinoma: A Systematic Review

Kakos¹,

Ziogas

Demiri

et al. 2022

Cancers

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Liver transplantation (LT) is the only potentially curative option for children with unresectable hepatocellular carcinoma (HCC). We performed a systematic review of the MEDLINE, Scopus, Cochrane Library, and Web of Science databases (end-of-search date: 31 July 2020). Our outcomes were overall survival (OS) and disease-free survival (DFS). We evaluated the effect of clinically relevant variables on outcomes using the Kaplan–Meier method and log-rank test. Sixty-seven studies reporting on 245 children undergoing LT for HCC were included. DFS data were available for 150 patients and the 1-, 3-, and 5-year DFS rates were 92.3%, 89.1%, and 84.5%, respectively. Sixty of the two hundred and thirty-eight patients (25.2%) died over a mean follow up of 46.8 ± 47.4 months. OS data were available for 222 patients and the 1-, 3-, and 5-year OS rates were 87.9%, 78.8%, and 74.3%, respectively. Although no difference was observed between children transplanted within vs. beyond Milan criteria (p = 0.15), superior OS was observed in children transplanted within vs. beyond UCSF criteria (p = 0.02). LT can yield favorable outcomes for pediatric HCC beyond Milan but not beyond UCSF criteria. Further research is required to determine appropriate LT selection criteria for pediatric HCC.

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Section: Introductionmentioning

confidence: 99%

Liver Transplantation for Pediatric Hepatocellular Carcinoma: A Systematic Review

Kakos¹,

Ziogas

Demiri

et al. 2022

Cancers

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“…33 However, studies have demonstrated that excellent recurrence-free survival can be achieved in pediatric patients with disease outside the Milan criteria in terms of size (>5 cm) or lesion number (>3), as long as no extrahepatic disease is present. [11][12][13]18 Several studies have also demonstrated acceptable outcomes after LT in children with evidence of macrovascular invasion, 12,13,18 despite vascular involvement being a strong predictor of posttransplant recurrence in adult patients. 34,35 Addition of sorafenib to chemotherapy regimens yielded only modest improvement (57% of patients had a partial response) in a small series.…”

Section: Discussionmentioning

confidence: 99%

“…Currently, however, no consensus criteria exist for pediatric-specific LT for HCC, but several reports suggest that eligibility may be expanded beyond Milan criteria used for adults with HCC. [10][11][12][13] Although LT has established roles in the management algorithms for advanced, unresectable disease in both pediatric HBL and HCC, current United States organ allocation policies differ for these groups of patients. No study to date has examined the waitlist and posttransplant outcomes of these patients in concert.…”

Section: Introductionmentioning

confidence: 99%

Waitlist mortality and post‐liver transplant outcomes of pediatric patients with hepatocellular carcinoma and hepatoblastoma in the United States

Ziogas

Matsuoka

et al. 2021

Pediatric Blood & Cancer

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Background: Liver transplantation (LT) is offered in cases of advanced disease for both pediatric patients with hepatoblastoma (HBL) and those with hepatocellular carcinoma (HCC). Current United States organ allocation priorities differ between the two groups. Methods:We retrospectively examined the waitlist and posttransplant outcomes of pediatric LT candidates with HBL and HCC using the United Network for Organ Sharing registry (February 2002 to September 2020). Results: Six hundred sixty-eight children with HBL and 95 children with HCC listed for first LT were identified. Patients with HBL were younger (p < .001), had lower laboratory Model for End-stage Liver Disease (MELD)/Pediatric End-stage Liver Disease (PELD) scores (p < .001), and had lesser proportion with encephalopathy (p = .01).Patients with HCC had an increased risk of waitlist mortality in univariable (unadjusted subdistribution hazard ratio [sHR] = 4.37, 95% confidence interval [CI], 2.01-9.51, p < .001) and multivariable competing risk regression (adjusted sHR = 3.08, 95% CI 1.13-8.37, p = .03) accounting for age and laboratory MELD/PELD score. Five hundred ninety-five children underwent LT for HBL and 76 for HCC. Patients transplanted for HBL had a significantly higher proportion with status 1B exception (71.3% vs. 7.9%, p < .001). No difference was observed in patient (unadjusted log-rank test, p = .52; adjusted hazard ratio [HR] = 0.77, 95% CI, 0.40-1.48, p = .43) or graft survival (unadjusted log-rank test, p = .93; adjusted HR = 0.74, 95% CI 0.42-1.33, p = .32) between HCC and HBL recipients. Conclusion:Waitlist mortality for pediatric LT candidates with HCC is significantly higher than for HBL, while posttransplant patient and graft survival are similar. This highlights an opportunity to improve equitable prioritization for children with HCC who may have reduced access to size-appropriate deceased donor organs and less effective bridge-to-transplant therapies.

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“…Of note, none of these are validated in pediatric HCC [86] . A general consensus is to offer LT for unresectable HCC patients and no extrahepatic disease [87] , while LT should be considered even for patients with HCC PRETEXT I or II in selected cases [88] .…”

Section: Indicationsmentioning

confidence: 99%

“…When studying treatment choice, a 21-patient series comparing outcomes for resection and chemotherapy vs. LT in pediatric HCC reveals a superior survival (72% vs. 40%) in the LT subgroup, pointing out the need for the early evaluation of transplantability of pediatric HCC patients in the treatment course [88] .…”

Section: Contraindicationsmentioning

confidence: 99%

A practical approach to pediatric liver transplantation in hepatoblastoma and hepatocellular carcinoma

Calinescu¹,

Hery²,

Goyet³

et al. 2021

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Progressively, as chemotherapy has become more effective, more children with liver malignancies are amenable to liver transplantation, and indications have expanded from a limited range of cases (mostly hepatoblastoma) to a range of other unresectable malignant liver tumors; as a result, more children with hepatocellular carcinoma are also now proposed to transplantation, even and often outside the Milan criteria, for a cure. Recent series have highlighted that patient and graft survivals after transplantation for hepatoblastoma and hepatocellular carcinoma have improved in the last decade. Although consensus has not yet been reached about transplantation as a possible cure for other tumor types than hepatoblastoma and hepatocellular carcinoma, liver transplantation, generally speaking, has become an important pillar in the management of pediatric liver malignancies. Remaining limitations and inquiries relate to patient selection (in term of selection criteria considering the risk of recurrence), the role and usefulness of chemotherapy after transplantation, or the best immunosuppression strategy to both protect renal function and improve outcome. Although some prospective studies are on the way regarding these aspects, more studies are needed to explore this rapidly changing aspect of care.

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Surgical management of pediatric hepatocellular carcinoma: An analysis of the National Cancer Database

Cited by 13 publications

References 20 publications

Liver Transplantation for Pediatric Hepatocellular Carcinoma: A Systematic Review

Liver Transplantation for Pediatric Hepatocellular Carcinoma: A Systematic Review

Waitlist mortality and post‐liver transplant outcomes of pediatric patients with hepatocellular carcinoma and hepatoblastoma in the United States

A practical approach to pediatric liver transplantation in hepatoblastoma and hepatocellular carcinoma

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